{"id":20283,"date":"2017-04-28T13:00:24","date_gmt":"2017-04-28T19:00:24","guid":{"rendered":"http:\/\/www.salud.carlosslim.org\/english2\/?p=20283"},"modified":"2017-04-27T14:37:09","modified_gmt":"2017-04-27T20:37:09","slug":"cell-gene-therapy-cures-bubble-baby-disease-in-phase-2-clinical-trials","status":"publish","type":"post","link":"http:\/\/www.salud.carlosslim.org\/english2\/cell-gene-therapy-cures-bubble-baby-disease-in-phase-2-clinical-trials\/","title":{"rendered":"Cell Gene Therapy Cures Bubble Baby Disease in Phase 2 Clinical Trials"},"content":{"rendered":"<p><strong><img loading=\"lazy\" decoding=\"async\" class=\"alignleft wp-image-20313\" src=\"http:\/\/www.salud.carlosslim.org\/english2\/wp-content\/uploads\/2017\/04\/baby-bubble-disease.jpg\" alt=\"\" height=\"130\" width=\"196\" srcset=\"http:\/\/www.salud.carlosslim.org\/english2\/wp-content\/uploads\/2017\/04\/baby-bubble-disease.jpg 600w, http:\/\/www.salud.carlosslim.org\/english2\/wp-content\/uploads\/2017\/04\/baby-bubble-disease-300x199.jpg 300w, http:\/\/www.salud.carlosslim.org\/english2\/wp-content\/uploads\/2017\/04\/baby-bubble-disease-250x166.jpg 250w\" sizes=\"auto, (max-width: 196px) 100vw, 196px\" \/>A stem cell gene therapy developed at University of California &#8211; Los Angeles (UCLA) shows effective for babies with bubble baby disease (or ADA-SCID), in phase 2 clinical trials<\/strong>.<\/p>\n<p><strong>Adenosine deaminase-deficiency (ADA) is a rare life-threatening inherited condition that damages the immune system and causes severe combined immunodeficiency (SCID). People with SCID lack virtually all immune protection from bacteria, viruses, and fungi<\/strong>. They are prone to repeated and persistent infections that can be very serious or life-threatening. <strong>Babies who born with this disease can die in the first year of life when untreated<\/strong>. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.<\/p>\n<p><strong>According to the results, all 9 babies who participated in the latest clinical trial were cured<\/strong>. However, <strong>another participant who was a teenager when he received the treatment showed no signs of recovery<\/strong>. <strong>So far, the stem cell gene therapy has restored the immune system of 30 out of 30 babies by using their own cells, during the different clinical trials<\/strong>.<\/p>\n<p>The UCLA <strong>researchers used a<\/strong> <strong>strategy to correct the ADA-SCID mutation by genetically modifying the patient\u2019s blood-forming stem cells<\/strong>, which can create all blood cell types. In the trial, blood stem cells were removed from each child\u2019s bone marrow; and then were corrected in the lab by inserting the gene responsible for making the adenosine deaminase enzyme. Finally, each child received a transplant of their own corrected blood stem cells.<\/p>\n<p>The <strong>fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients<\/strong>, before their bodies lose the ability to restore the immune system.<\/p>\n<p>The next step is obtaining approval from the US Food and Drug Administration (FDA) to be able to treat children with ADA-SCID.<\/p>\n<p>&nbsp;<\/p>\n<p><strong>Source: <\/strong><em>UCLA Newsroom<\/em><\/p>\n","protected":false},"excerpt":{"rendered":"<p>A stem cell gene therapy developed at University of California &#8211; Los Angeles (UCLA) shows effective for babies with bubble baby disease (or ADA-SCID), in phase 2 clinical trials. Adenosine deaminase-deficiency (ADA) is a rare life-threatening inherited condition that damages the immune system and causes severe combined immunodeficiency (SCID). People with SCID lack virtually all &#8230;<\/p>\n","protected":false},"author":7,"featured_media":20313,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"ngg_post_thumbnail":0,"footnotes":""},"categories":[3],"tags":[15,12],"class_list":["post-20283","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-actualidad-en-la-salud","tag-casalud","tag-genomica","cat-3-id","has_thumb"],"_links":{"self":[{"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/posts\/20283","targetHints":{"allow":["GET"]}}],"collection":[{"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/users\/7"}],"replies":[{"embeddable":true,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/comments?post=20283"}],"version-history":[{"count":4,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/posts\/20283\/revisions"}],"predecessor-version":[{"id":20314,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/posts\/20283\/revisions\/20314"}],"wp:featuredmedia":[{"embeddable":true,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/media\/20313"}],"wp:attachment":[{"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/media?parent=20283"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/categories?post=20283"},{"taxonomy":"post_tag","embeddable":true,"href":"http:\/\/www.salud.carlosslim.org\/english2\/wp-json\/wp\/v2\/tags?post=20283"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}